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At my first gene therapy conference, which I attended in 2017, digital PCR was almost not mentioned. Today, digital PCR has become an indispensable tool in gene therapy research and manufacturing. On April 23rd, I am hosting a webinar with Stilla Technologies about The Impact of Digital PCR on Quantification in Biopharma Research And Manufacturing. You can register here: https://hubs.la/Q02rjMDP0 Hope to see you there! #biopharma #genetherapy #webinar

Pharmaceuticals are getting Crispier How? For a long time in busy labs of scientists with high IQs, every gene editing required specific DNA cutting enzymes. Once upon a time, they stumbled upon CRISPR-Cas9, a gene-editing tool that only requires a custom guide RNA molecule to find its target site. This allowed scientists to turn their frown, upside down. CRISPR-Cas9 has made waves in late 2023 with the approval of Casgevy, a cell-based gene therapy, approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Now, Vertex. Know more at: https://lnkd.in/eAW4TyVh #geneediting #biologics #fda

Are you interested in the latest advances and challenges in gene therapy development? The new book from Yanmei Lu and Boris Gorovits covers everything from translational biomarkers and bioanalytical methods to regulatory issues and companion diagnostics for gene therapy products. Expert contributors with extensive experience in the field provide insights from real-world clinical examples. Whether you are a researcher, developer, or student in the biopharmaceutical industry, this book will help you navigate the complex and exciting world of gene therapy. https://lnkd.in/e5bnG9tq #genetherapy #biopharma #drugdevelopment #biomarkers #companiondiagnostics

Process validation in cell and gene therapy manufacturing is a complex undertaking, but strong control strategy principles can ease the journey to approval. In the snippet below, Monika Pema, Senior Manager of MSAT at AGC Biologics, outlines four critical aspects to be considered in the definition of your control strategy during Stage 1. View the full webinar to learn more key considerations for each step of the cell and gene therapy commercialization journey, now available on-demand: https://hubs.li/Q02GhFR20 #CellAndGeneTherapy #GeneTherapy #Biologics #GeneTherapyDevelopment #Biopharma #BiotechWebinar

e-Lesson of the day: 🔎 ATMP MANUFACTURING - CELL AND GENE THERAPIES This Digital Learning Module is designed for personnel who require an overview of the nature and function of cell and gene therapy products, their modes of manufacture (with a particular focus on autologous and allogeneic therapies), and the associated technical challenges and regulatory requirements. Follow SKILLPAD - digital learning for life sciences on LinkedIn at https://lnkd.in/ejd-z5Am for more news and updates. ATMP MANUFACTURING - CELL AND GENE THERAPIES covers these Learning Objectives: ✅ Define “Advanced Therapy Medicinal Product” (ATMP) ✅ Explain the difference between ATMPs and ‘traditional’ biopharmaceutical products ✅ Explain the difference between gene and cell therapies ✅ Describe the biological functionality of cell therapies ✅ Describe the biological functionality of gene therapies ✅ Distinguish between autologous and allogeneic therapeutic approaches ✅ List the main steps in manufacturing autologous therapies ✅ List the main steps involved in manufacturing allogeneic therapies ✅ Describe the challenges associated with ATMP manufacturing, and in general terms how these challenges might be overcome Description and screenshots here: https://lnkd.in/e96Km6KV Schedule a DEMO by contacting [email protected] #pharma #biotech #biologics #biopharma #biopharmaceuticals #lifesciences #genetherapies #atmp

#Webinar now available on demand! #QuadplexdPCR as a tool to analyze #genomeintegrity and heterogeneity in gene therapy drug products The rapid growth of the #genetherapy field necessitates product-specific analytics, as critical quality attributes do not consistently correlate with drug potency or safety. It is hypothesized that the heterogeneity of packaged genomes contributes to this inconsistency. Ryan Massopust (Scientist II, Analytical Development, Asklepios BioPharmaceutical, Inc. (AskBio)) explores a quadplex digital PCR (#dPCR) assay, employing four primer probe sets that span the genome from promoter to poly(A) signal sequence, that was developed to investigate this hypothesis. Watch this webinar at your convenience below:

Are you looking to increase the characterization and understanding of your gene therapy products? Join us at the 3-day "Gene Therapy CMC and Manufacturing track" at the Bioprocessing Summit, August 19-22, at Sheraton Boston, to unlock the potential of advanced analytics in ensuring product quality and consistency. https://lnkd.in/dGbQzqv Reasons to Attend: - Navigate comparability challenges in gene therapy with Phillip Ramsey from Sangamo Therapeutics, Inc. and Xiaohui Lu, Ultragenyx Pharmaceutical - Optimize your analytics for AAV characterization with santoshkumar khatwani L. Khatwani, Sangamo Therapeutics - Investigate cutting-edge mass spectrometry techniques with @jonathan Bones, NIBRT National Institute for Bioprocessing Research and Training - Benchmark against the latest RT-MALS applications in biologics and vaccines from Daniel Some, Waters | Waters | Wyatt Technology - Evaluate high-throughput AAV quantification methods with Xueyuan Liu, Childrens Hospital of Philadelphia This session forms part of the "Gene Therapy CMC and Analytics track at ‘Bioprocessing Summit’, and is one of 14 full conference programs at the 2024 Bioprocessing Summit in Boston on August 19-22. These programs cover a wide range of themes, including AI and Digitalization, Cell Therapy, Upstream and Downstream Processing. Join us at the Bioprocessing Summit for a ground-breaking event featuring 1,500 biomanufacturing experts across: 14 𝐂𝐨𝐧𝐟𝐞𝐫𝐞𝐧𝐜𝐞 𝐓𝐫𝐚𝐜𝐤𝐬 including a new stream on "AI and Digitalization" 300 𝐏𝐫𝐞𝐬𝐞𝐧𝐭𝐚𝐭𝐢𝐨𝐧𝐬 across the spectrum of biologics, gene, cell, and mRNA therapies 90 𝐄𝐱𝐡𝐢𝐛𝐢𝐭𝐨𝐫𝐬 showcasing innovative bioprocessing technologies and services 100 𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡 𝐏𝐨𝐬𝐭𝐞𝐫𝐬 revealing the latest in biomanufacturing and analytics 𝐓𝐰𝐨 𝐏𝐥𝐞𝐧𝐚𝐫𝐲 𝐒𝐞𝐬𝐬𝐢𝐨𝐧𝐬 tackling the current and future challenges in bioprocessing #chibioprocessing

Gene&Cell Therapy >> #ADA24: Fractyl pitches potential of GLP-1 gene therapy, but pushes clinical trials back to 2025: A small biotech company is developing a one-time gene therapy that could turn the pancreas into its own GLP-1 drug factory and eliminate the need for frequent injections of medicines like Ozempic or Wegovy. Fractyl Health previously showed that an injection of its treatment, delivered in viral vectors injected directly into the liver, caused obese mice to lose nearly 25% of their body weight in just two weeks. At a scientific conference on Sunday, the company said that mice who got a GLP-1 drug for four weeks maintained their 22% weight loss four weeks later when they switched to the gene therapy. The updated data were presented at the American Diabetes Association’s scientific sessions in Florida. In an interview with Endpoints News ahead of the conference, Fractyl CEO Harith Rajagopalan said that the yo-yo-ing weight loss and gain seen in people who go on and off GLP-1 drugs emphasizes the need for one-and-done alternatives. “The question is shifting from how do you lose weight to how do you keep it off,” Rajagopalan said. “If the effects wear off, you are talking about limited benefit for society. But if you can make those effects permanent, you can imagine what a post-obesity society looks like.” Last year at the diabetes conference, Fractyl announced plans to begin testing its gene therapy in both diabetes and obesity patients in 2024. But that ambitious timeline has slipped — as has the company’s stock, which is down about 70% since its February IPO. Rajagopalan said the company is now planning a clinical trial in patients with diabetes for 2025. The results will inform a future trial testing a slightly different gene therapy design in people with obesity, but Rajagopalan wouldn’t say when that trial might be. Longer-term studies in animals are also underway, he said. While there’s been ongoing interest from drugmakers about how to decrease the frequency of dosing for weight loss drugs and to make their effects last longer, a permanent treatment would likely raise many of its own questions. The drugs have been associated with side effects such as muscle loss, which could become a particular issue as patients age and become more frail, for example. Novo Nordisk recently opened a research center outside of Boston focused on genetic medicines. Its leaders told Endpoints in February that it is working on genetic medicines that could complement its weight loss drugs but haven’t disclosed interest in making a gene therapy version of Wegovy. And Eli Lilly, which is also opening a genetics-focused research hub in Boston, has also remained mum on the prospects for a GLP-1 gene therapy. Rajagopalan says that the company’s endoscopic procedure for shuttling the AAV gene therapy directly into the pancreas is… #lucidquest #genetherapy #celltherapy

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> #ADA24: Fractyl pitches potential of GLP-1 gene therapy, but pushes clinical trials back to 2025: A small biotech company is developing a one-time gene therapy that could turn the pancreas into its own GLP-1 drug factory and eliminate the need for frequent injections of medicines like Ozempic or Wegovy. Fractyl Health previously showed that an injection of its treatment, delivered in viral vectors injected directly into the liver, caused obese mice to lose nearly 25% of their body weight in just two weeks. At a scientific conference on Sunday, the company said that mice who got a GLP-1 drug for four weeks maintained their 22% weight loss four weeks later when they switched to the gene therapy. The updated data were presented at the American Diabetes Association’s scientific sessions in Florida. In an interview with Endpoints News ahead of the conference, Fractyl CEO Harith Rajagopalan said that the yo-yo-ing weight loss and gain seen in people who go on and off GLP-1 drugs emphasizes the need for one-and-done alternatives. “The question is shifting from how do you lose weight to how do you keep it off,” Rajagopalan said. “If the effects wear off, you are talking about limited benefit for society. But if you can make those effects permanent, you can imagine what a post-obesity society looks like.” Last year at the diabetes conference, Fractyl announced plans to begin testing its gene therapy in both diabetes and obesity patients in 2024. But that ambitious timeline has slipped — as has the company’s stock, which is down about 70% since its February IPO. Rajagopalan said the company is now planning a clinical trial in patients with diabetes for 2025. The results will inform a future trial testing a slightly different gene therapy design in people with obesity, but Rajagopalan wouldn’t say when that trial might be. Longer-term studies in animals are also underway, he said. While there’s been ongoing interest from drugmakers about how to decrease the frequency of dosing for weight loss drugs and to make their effects last longer, a permanent treatment would likely raise many of its own questions. The drugs have been associated with side effects such as muscle loss, which could become a particular issue as patients age and become more frail, for example. Novo Nordisk recently opened a research center outside of Boston focused on genetic medicines. Its leaders told Endpoints in February that it is working on genetic medicines that could complement its weight loss drugs but haven’t disclosed interest in making a gene therapy version of Wegovy. And Eli Lilly, which is also opening a genetics-focused research hub in Boston, has also remained mum on the prospects for a GLP-1 gene therapy. Rajagopalan says that the company’s endoscopic procedure for shuttling… #lucidquest #genetherapy #celltherapy

Our recent webinar with AAPS is now available to view on demand! American Association of Pharmaceutical Scientists (AAPS) | @aapscomms This webinar introduces FIM and discusses how it can be used to monitor subvisible particles in gene therapy drug products. We also present a case study in which FIM was used to analyze AAV formulations during an accelerated stability study. Applications for monitoring subvisible particles in other types of gene therapies are also discussed. https://lnkd.in/eDaPg4YM